Division of Oncology, Department of Internal Medicine, Incheon St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Incheon, Korea.
Division of Hematology-Oncology, Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea.
Korean J Intern Med. 2021 May;36(3):668-678. doi: 10.3904/kjim.2019.367. Epub 2020 Aug 14.
BACKGROUND/AIMS: Waldenström macroglobulinemia (WM) is a rare lymphoproliferative disorder that usually follows an indolent clinical course. However, some patients show an aggressive clinical course leading to death. We explored the risk factors predicting poor prognosis in WM patients.
We retrospectively analyzed 47 patients diagnosed with WM between 2000 and 2018 to explore risk factors predicting poor prognosis using various clinical and laboratory parameters and risk models including the International Prognostic Staging System for WM (IPSS-WM).
Over a median follow-up duration of 80.4 months, 29 patients died. The main causes of death were disease progression, organ failure related to amyloidosis, and infection. The median overall survival (OS) was 55.1 months, and 14 patients, including three with amyloidosis, died within 2 years. Serum β2-microglobulin level higher than 4 mg/dL was significantly associated with poor OS. Accordingly, the IPSS-WM showed a significant association with poor prognosis compared with other risk models, and the low-risk group had better OS than intermediate- and high-risk groups. In the retrospective analysis using the results of targeted sequencing in two cases representing good and bad prognosis, different patterns of mutation profiles were observed, including mutations of MYD88, TP53, ARID1A, and JAK2 in a refractory case.
Serum β2-microglobulin could be a single biomarker strongly predictive of poor survival of WM patients, and the low-risk group of the IPSS-WM risk model including serum β2-microglobulin has better prognostic value than other risk models. Mutation analysis also might provide additional information to predict high-risk patients.
背景/目的：华氏巨球蛋白血症（WM）是一种罕见的淋巴增殖性疾病，通常表现为惰性临床病程。然而，一些患者表现出侵袭性临床病程，导致死亡。我们探讨了预测 WM 患者不良预后的危险因素。
我们回顾性分析了 2000 年至 2018 年间诊断为 WM 的 47 例患者，通过各种临床和实验室参数以及风险模型（包括国际 WM 预后分期系统（IPSS-WM））来探讨预测不良预后的危险因素。
在中位随访 80.4 个月期间，29 例患者死亡。死亡的主要原因是疾病进展、淀粉样变性相关的器官衰竭和感染。中位总生存期（OS）为 55.1 个月，14 例患者（包括 3 例有淀粉样变性）在 2 年内死亡。血清β2-微球蛋白水平高于 4mg/dL 与不良 OS 显著相关。因此，与其他风险模型相比，IPSS-WM 与不良预后显著相关，低危组的 OS 优于中高危组。在对代表预后良好和不良的 2 例患者的靶向测序结果进行回顾性分析时，观察到不同的突变谱模式，包括难治性病例中 MYD88、TP53、ARID1A 和 JAK2 的突变。
血清β2-微球蛋白可能是预测 WM 患者生存不良的单一强生物标志物，包括血清β2-微球蛋白的 IPSS-WM 风险模型的低危组比其他风险模型具有更好的预后价值。突变分析也可能提供额外的信息来预测高危患者。
